Gene Therapy Guidances From US FDA Coming In About 6 Weeks, Gottlieb Says
Potential accelerated approval endpoints will be addressed; FDA Commissioner expects gene therapy to become a 'backbone' treatment as antibodies are now.
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Although a sponsor may conclude that a long-term follow-up protocol is not necessary for a gene therapy, FDA may have a different interpretation, and the agency won't let studies start if that's the case, draft gudiance states.
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